Phase 2/3 testing of osivelotor in adults and adolescents with sickle cell disease.

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This multicenter, Phase 2/3 program gives oral osivelotor to adolescents and adults with sickle cell disease to define an optimal dose, compare efficacy with placebo over 48 weeks, and offer a long-term open-label extension for safety and blood response monitoring. Part A is a dose-finding phase in adults to establish safety, tolerability and pharmacokinetics/pharmacodynamics. Part B randomizes adults and adolescents to osivelotor or placebo to measure clinical outcomes including vaso-occlusive crises over 48 weeks. The Open-Label Extension follows participants who completed Part B to collect longer-term safety and hematologic data.

Why it matters

Who can join: the full rules

Inclusion Criteria:

Part A and Part B:

* Male or female with SCD, HbSS and HbSB-zero
* Participants with Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL during Screening and considered stable by the Investigator.
* For participants taking hydroxyurea and/or L-glutamine, the dose must be stable for at least 90 days prior to signing the ICF or assent and with no anticipated need for dose adjustments during the study in the opinion of the Investigator.

Part B:

* Participants with SCD ages 12 years and older, inclusive at screening.
* Participants with more than or equal to 2 and ≤ 10 VOCs within 12 months of Screening.

OLE:

\- Participants who have completed the Part B successfully will be eligible.

Exclusion Criteria:

Part A and Part B:

* Participants who had more than 10 VOC within 12 months of screening
* Female participant who is breastfeeding or pregnant
* Participants who receive RBC transfusion therapy regularly or received an RBC transfusion ---for any reason within 90 days of Day 1
* Participants hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF or anytime during the screening period.
* Participants in Sub-Saharan Africa or who have relocated from Sub-Saharan Africa within 6 months.

OLE:

* Have any unresolved clinically significant adverse event, laboratory abnormality, or safety finding from Part B that, in the opinion of the Investigator, increases the risk of study drug administration.
* Met permanent treatment discontinuation criteria during Part B.
* Have withdrawn consent or are unable to comply with study procedure

Where it's running

• AMR Clinical
  Mobile, Alabama, United States Recruiting
• Edward Jenner Research Group LLC.
  Plantation, Florida, United States Recruiting
• Pediatric Hematology / Oncology a division of Kidz Medical services
  West Palm Beach, Florida, United States Recruiting
• St. Mary's Medical Center
  West Palm Beach, Florida, United States Recruiting
• Children's Center for Advanced Pediatrics
  Atlanta, Georgia, United States Recruiting
• University of Illinois at Chicago Clinical Research Center
  Chicago, Illinois, United States Recruiting

+30 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Pfizer Pfizer CT.gov Call Center
• Email: ClinicalTrials.gov_Inquiries@pfizer.com
• Phone: 1-800-718-1021

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.